The neuronal ceroid lipofuscinoses (NCL) are severe neurodegenerative lysosomal storage
disorders of childhood, characterized by accumulation of autofluorescent ceroid …

Neuronal ceroid lipofuscinoses (NCL) comprise a group of inherited lysosomal disorders
with variable age of onset, characterized by lysosomal accumulation of autofluorescent …

Despite the identification of a large number of disease-causing genes in recent years, it is
still unclear what disease mechanisms operate in the neuronal ceroid lipofuscinoses (NCLs …

Background One of the most important steps taken by Beyond Batten Disease Foundation in
our quest to cure juvenile Batten (CLN3) disease is to understand the State of the Science …

The neuronal ceroid lipofuscinoses (NCL) are a group of disorders defined by shared
clinical and pathological features, including seizures and progressive decline in vision …

Linking bioactive compounds to their cellular targets is a central challenge in chemical
biology. Here we report the mode of action of theonellamides, bicyclic peptides derived from …

Neuronal ceroid lipofuscinoses are a group of fatal progressive neurodegenerative diseases
predominantly affecting children. Identification of mutations that cause neuronal ceroid …

Tropomyosin (Tm) is a conserved dimeric coiled-coil protein, which forms polymers that curl
around actin filaments in order to regulate actomyosin function. Acetylation of the Tm N …

The CLN3 gene was identified over two decades ago, but the primary function of the CLN3
protein remains unknown. Recessive inheritance of loss of function mutations in CLN3 are …

The neuronal ceroid lipofuscinoses (NCLs, Batten disease) are a group of inherited
childhood‐onset neurodegenerative disorders characterized by the lysosomal accumulation …